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Epic Bio Presents New Data Highlighting Potential of EPI-321 for FSHD and Epigenetic Editing Platform Leadership at ASGCT Annual Meeting

– Across robust slate of presentations, company highlights compelling new data and strong differentiation –

SOUTH SAN FRANCISCO, Calif. — April 23, 2024 — Epic Bio, a leading epigenetic editing company that plans to have its FSHD program enter the clinic this year, today shared details of data being presented at the Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 7-11, 2024, in Baltimore, Maryland. Across three oral presentations and three poster presentations, Epic Bio will present new data demonstrating the differentiation of the company’s epigenetic editing platform and the promise of lead candidate EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

“We’re proud of the robust presence Epic Bio will have at this year’s ASGCT, reflecting the high degree of excitement and interest in epigenetic editing we’re seeing across the biopharma industry as well as Epic Bio’s leading position within this space,” said Amber Salzman, Ph.D., chief executive officer of Epic Bio. “We are laser-focused on translating these capabilities into medicines that can make a meaningful difference for patients, starting with our lead candidate EPI-321 for FSHD, which will enter the clinic later this year.”

Best-in-class epigenetic activators

In an oral presentation at the conference, the company will present new data demonstrating the best-in-class profile of its proprietary epigenetic activators. While epigenetic gene silencing has been widely successful, robust and persistent activation of genes remains a significant challenge, and an area where epigenetic editing may offer the greatest therapeutic potential compared to other genetic medicines.

New data on Epic Bio’s epigenetic activators show that they can produce long-term activation of IL-21 — an interleukin crucial for enhancing CAR-T function — at levels far exceeding those produced by one of today’s gold-standard activators, VPR.

A second study evaluated the use of proprietary epigenetic activators to boost transcription of the LDL Receptor (LDLR) gene. Mutations in one copy of LDLR are the most common cause of familial hypercholesterolemia, and thus epigenetic activation of LDLR is a highly promising potential treatment approach. In liver cells, Epic Bio’s epigenetic activators produced sustained LDLR expression. Critically, this activation was shown to persist for 70 days though the activator itself was only transiently expressed. Furthermore, in a humanized mouse model the proprietary activators boosted LDLR expression by twofold, sustained for up to 5 weeks, even though the activator was transiently delivered — far exceeding all previously reported outcomes of in vivo testing of a CRISPR-based epigenetic activator. These insights will inform Epic Bio’s ongoing preclinical efforts to develop an epigenetic editing therapy for heterozygous familial hypercholesterolemia (HeFH) utilizing one or more mechanisms of action.

Establishing the standard for off-target characterization in epigenetic editing

In a second oral presentation, Epic Bio is presenting the design and results of its whole-genome off-target assessment for the company’s lead candidate, EPI-321. As a leader in the field of epigenetic editing, Epic Bio has done pioneering work to craft a comprehensive off-target assessment platform to ensure the safety of epigenetic editing therapies for human testing. This analysis, designed with inputs from regulators in the U.S. and elsewhere, leverages genome-wide methylation profiling, genome-wide transcriptomic profiling, in silico prediction, and targeted validation assays across various model systems in order to robustly identify putative off-target effects.

In the application of this off-target assessment to EPI-321, the company found that there are no direct off-target events caused by EPI-321 treatment, supporting the clinical initiation for EPI-321 later this year.

A tool to expand access to hypercompact Cas proteins

A third oral presentation at ASGCT spotlights a novel, freely available tool recently developed by Epic Bio to assist researchers who wish to use the hypercompact dCasMINI protein for epigenetic editing. The small size of dCasMINI — less than half the size of dCas9 — makes it ideally suited for single-vector delivery, regardless of cargo capacity. However, unlike Cas9 or Cas12a, there are no available computational tools for designing CasMINI guides, which presents a significant barrier to its ease-of-use by academic and industry researchers and drug developers.

Epic Bio developed a web-based application to support design, selection, and comprehensive off-target prediction of guide RNAs for epigenetic activation and suppression (CRISPRa/i) using the catalytically inactivated dCasMINI CRISPR-Cas system against any gene in the human genome. The tool should enable researchers to easily employ dCasMINI in their investigations, and thereby foster continued scientific and medical innovation.

Poster presentations
In addition to the above oral presentations, the company will present posters on:
• The complete nonclinical data package for lead candidate EPI-321, including supplemented efficacy and safety data since the company’s oral presentation at ASGCT last year, along with new data on functional measures using 3-D engineered human muscle tissue.
• Details of a generative AI approach to design de novo transcriptional activators, and
• Results of work to characterize and optimize Epic Bio’s proprietary Cas proteins.

Posters will be made available on the Science section of the Epic Bio website.

About EPI-321
EPI-321 is an investigational epigenetic therapy that aims to address the underlying molecular mechanisms of FSHD by restoring methylation to the D4Z4 region of chromosome 4 and halting toxic expression of the DUX4 gene. EPI-321 is delivered to muscle tissue within a single AAV vector (AAVrh74) which has been clinically validated for muscle delivery. Preclinical studies on EPI-321 have demonstrated its ability to robustly suppress pathological expression of the DUX4 gene and reduce muscle cell death.

About Epic Bio
Epic Bio is a leading epigenetic editing company, leveraging the power of CRISPR without cutting DNA. The company’s proprietary Gene Expression Modulation System (GEMS) includes the smallest Cas protein known to work in human cells, enabling in vivo or ex vivo delivery via a single viral vector. Epic plans to begin dosing patients in a clinical trial of its lead program — EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) — in 2024; additional programs seek to address alpha-1 antitrypsin deficiency (A1AD), heterozygous familial hypercholesterolemia (HeFH), and retinitis pigmentosa (RP). Visit for more information or follow us on X and LinkedIn.

Investor Contact
Shawn M. Cox
Epic Bio
Manager, Investor Relations, and Corporate Communications

Media Contact
Lisa Raffensperger
Ten Bridge Communications
(617) 903-8783

The latest from Epic

16 Nov

"The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD), the most common form of muscular dystrophy in adults. Read more here: "

7 Nov

"We are pleased to share that Epic Bio CEO Dr. Amber Salzman has been invited to present at the 2023 Stiefel Healthcare Conference. Read more here: "

31 Oct

"@Epicbiotech partners with @KitePharma to extend the potential application of Epic’s platform for cancer treatment. Epic’s approach is promising for in vivo medicines and may enable next-generation cell therapies #epigenetics #CellTherapy #Cancer "